ThrombocytosisPhase 2NCT02125318
A Study of Anagrelide Controlled Release (GALE-401) in Patients With High Platelet Counts Due to Bone Marrow Disorders
A Phase 2 study testing Anagrelide CR in people with Thrombocytosis and Myeloproliferative Neoplasms, run by Galena Biopharma, Inc. at 11 sites in United States.
Trial summary
Anagrelide is a drug that has been shown to slow down how fast platelets are made in the bone marrow, and has been approved by the FDA for treating high platelets counts in patients with bone marrow disorders. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytosis, Myeloproliferative Neoplasms
- What is being tested?
- Anagrelide CR
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 11 sitesUnited States
- Study dates
- May 2014to May 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Provide written informed consent prior to any study specific procedure
- Male or female patients aged ≥ 18 years
- Diagnosis of a myeloproliferative neoplasm (i.e., chronic myelogenous leukemia (CML), polycythemia vera (PV), primary myelofibrosis (PMF), essential thrombocythemia (ET)) as determined by the treating physician, such as based on the 2008 World Health Organization (WHO) classification of myeloid malignancies
- Baseline platelet count ≥600 x 10e9/L as determined on two occasions at least 14 days apart prior to the first dose of study drug
- Requirement for platelet reduction therapy as assessed by the Investigator
- Currently not receiving therapy specifically intended to reduce platelet counts
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- For patients with ET, prior platelet lowering therapy (e.g., hydroxyurea, anagrelide or interferon) may not be administered within 2 weeks prior to the first dose of study drug.
- For patients with MPN diagnoses other than ET, concurrent anti-MPN treatment is permitted provided that the treatment has been administered at stable doses for at least 4 weeks prior to the first dose of study drug. Examples of permitted medications include but are not limited to hydroxyurea for PV, ruxolitinib for MF, and imatinib for CML. All patients must have discontinued anagrelide at least 2 weeks prior to the first dose of study drug.
- EXCEPTION: busulfan, melphalan and phosphate P-32 must have been discontinued at least 4 weeks prior to the first dose of study drug.
- Adequate hepatic function defined as bilirubin ≤1.5 x ULN, INR ≤1.5 x ULN, albumin >3.5 g/dL, ALT < 3.0 x ULN, AST < 3.0 x ULN
- If female, must be of non-childbearing potential, i.e. post- menopausal (defined as > 12 months since last menstrual period) or surgically sterilized (i.e. tubal ligation or hysterectomy at least 6 months prior to screening) or, if of childbearing potential, must not be pregnant or nursing
- Males and females of child bearing must agree to use an acceptable form of birth control until 28 days following the last dose of study drug
Who cannot join
- Other MPN diagnoses not specifically included above: Chronic neutrophilic leukemia, chronic eosinophilic leukemia, mastocytosis, and unclassifiable MPNs
- Previously found to be refractory to anagrelide therapy (i.e., failure to achieve a platelet count <600 x 10e9/L for reasons other than anagrelide-related toxicity)
- History of coronary artery disease requiring a revascularization procedure within 3 months prior to screening
- Left bundle branch block or sustained ventricular tachycardia (>30 seconds) evident on 12-lead ECG at screening
- Tachycardia defined as resting heart rate >100 bpm at screening
- Unstable angina (characterized by increasingly frequent episodes with modest exertion or at rest, worsening severity, or prolonged duration) within 3 months prior to screening
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- Transient ischemic attack (TIA) or stroke within 3 months prior to screening
- Unstable or clinically significant concurrent medical condition that would, in the opinion of the Investigator, jeopardize the safety of a subject and/or their compliance with the protocol.
- Current alcohol or drug abuse, or a significant medical condition that, in the opinion of the Investigator, may impair compliance with the requirements of the protocol
- History of allergic hypersensitivity to anagrelide or any component of its formulations
- Administration of Type 3 phosphodiesterase (PDE3) inhibitors (e.g., inamrinone, cilostazol, milrinone) within 2 weeks prior to initiating study treatment
- Administration of any investigational product within 4 weeks prior to initiating study treatment
- History of intolerance of other PDE3 inhibitors
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Innovative Medical Research of South Florida, Inc.
Aventura, Florida, 33180
East Valley Hematology and Oncology Medical Group
Burbank, California, 91505
California Cancer Associates for Research & Excellence (cCARE)
Encinitas, California, 92024
California Cancer Associates for Research & Excellence (cCARE)
Escondido, California, 92025
California Cancer Associates For Research and Excellence
Fresno, California, 93720
Gettysburg Cancer Center
Hillsdale, Pennsylvania, 17325
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Cancer Care Centers of South Texas
New Braunfels, Texas, 78130
Cancer Care Centers of South Texas
San Antonio, Texas, 78229
Cancer Center of Kansas
Wichita, Kansas, 67214
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27157
About this study
Official title: A Phase 2, Open Label Efficacy and Safety Study of Anagrelide Controlled Release (CR) in Subjects With Thrombocytosis Secondary to Essential Thrombocythemia and Other Myeloproliferative Neoplasms (MPN)
Anagrelide is a drug that has been shown to slow down how fast platelets are made in the bone marrow, and has been approved by the FDA for treating high platelets counts in patients with bone marrow disorders.
Anagrelide Controlled Release ("CR") is a new preparation of anagrelide that is made to dissolve more slowly than currently marketed versions of this drug. Because of this, the anagrelide is taken up into the blood more slowly. Researchers think that this slower release of the drug could help to lower side effects that might be caused by high blood levels when the drug dissolves as quickly as it does with the currently marketed product.
The main purposes of this study are to see how well Anagrelide CR can control platelet counts in patients with high platelet levels, to see what kind of side effects it causes, and to measure blood levels of the drug.
Read the detailed description
This is an open-label, single-arm, multicenter, Phase 2 study of anagrelide CR in subjects with an MPN-related thrombocytosis. Eligible subjects will include those who have not been previously treated for thrombocytosis or have not received platelet-lowering therapy for at least 2 weeks prior to study treatment. Each subject will receive anagrelide CR at a starting dose of 0.5 mg b.i.d. (1.0 mg/day), and is anticipated to continue study treatment for at least 24 weeks. Subjects who have achieved clinical benefit in the opinion of the Investigator and who are tolerating the study drug may continue study treatment until they develop unacceptable toxicity or other discontinuation criteria have been met.
The primary efficacy endpoint will be the proportion of subjects who achieve a platelet response (CR or PR). The safety and tolerability of study treatment will be assessed by the frequency and severity of adverse events as determined by NCI Common Terminology Criteria for Adverse Events (CTCAE) v 4.03. The PK profile of anagrelide CR will be assessed at the initial (0.5mg b.i.d.) and final titrated dose levels.
- Conditions
- Thrombocytosis, Myeloproliferative Neoplasms
- Being tested
- Anagrelide CR
- Keywords
- myeloproliferative neoplasm, chronic myelogenous leukemia, polycythemia vera, primary myelofibrosis, essential thrombocythemia, anagrelide, thrombocytosis, GALE-401
- Medical subject headings
- Thrombocytosis, Myeloproliferative Disorders, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Polycythemia Vera, Primary Myelofibrosis, Thrombocythemia, Essential
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anagrelide CRDrug
Also called GALE-401
Starting dose of 0.5 mg b.i.d. (1.0 mg/day) titrated at weekly intervals, on an individual basis, to achieve the lowest dose required to achieve and maintain a target platelet count of 150 - 400 x 10e9/L, tolerability permitting. The dose will be increased at weekly intervals in steps not exceeding 0.5 mg/day; the rate of dose titration may be reduced (i.e., up to once every 2 weeks) at the discretion of the Investigator.
| Study group | Role | What they receive |
|---|---|---|
| Anagrelide CR (GALE-401) | Experimental | Not described Receives: Drug: Anagrelide CR |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet Response
The primary efficacy endpoint will be the proportion of subjects who achieve a platelet response (complete or partial response), with response defined according to the following criteria:
- Complete response (CR): platelet count of ≤400 x 10e9/L maintained for at least 4 weeks
- Partial response (PR): a platelet count of ≤600 x 10e9/L or a ≥50% reduction from baseline and maintenance of the reduction for at least 4 weeks
- Nonresponse: failure to meet CR or PR criteria
Measured: Up to 24 weeks
Secondary outcomes (2)
Number of subjects with adverse events
Frequency and severity of adverse events as determined by NCI CTCAE (v 4.03).
Measured: Throughout study treatment (expected average of 12 months)
Plasma concentrations of anagrelide
Monitor anagrelide levels during dose titration and assess pharmacokinetic profile at starting and final dose levels.
Measured: Up to 13 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2014 (estimated)
- Study startMay 2014
- Primary completionMay 2016
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ThrombocytosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Galena Biopharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02125318
- Sponsor's protocol ID
- GALE-401-201
- Status last verified
- Feb 2017
- First submitted
- 23 Apr 2014
- Last updated
- 27 Feb 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open Label Efficacy and Safety Study of Anagrelide Controlled Release (CR) in Subjects With Thrombocytosis Secondary to Essential Thrombocythemia and Other Myeloproliferative Neoplasms (MPN)
- ClinicalTrials.gov ID
- NCT02125318
- Acronym / sponsor ID
- GALE-401-201
- Conditions
- Thrombocytosis, Myeloproliferative Neoplasms
- Keywords
- myeloproliferative neoplasm, chronic myelogenous leukemia, polycythemia vera, primary myelofibrosis, essential thrombocythemia, anagrelide, thrombocytosis, GALE-401
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Galena Biopharma, Inc.Industry (pharma, biotech, devices)
- Study start
- May 2014
- Primary completion
- May 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 29 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Anagrelide is a drug that has been shown to slow down how fast platelets are made in the bone marrow, and has been approved by the FDA for treating high platelets counts in patients with bone marrow disorders.
Anagrelide Controlled Release ("CR") is a new preparation of anagrelide that is made to dissolve more slowly than currently marketed versions of this drug. Because of this, the anagrelide is taken up into the blood more slowly. Researchers think that this slower release of the drug could help to lower side effects that might be caused by high blood levels when the drug dissolves as quickly as it does with the currently marketed product.
The main purposes of this study are to see how well Anagrelide CR can control platelet counts in patients with high platelet levels, to see what kind of side effects it causes, and to measure blood levels of the drug.
Detailed description
This is an open-label, single-arm, multicenter, Phase 2 study of anagrelide CR in subjects with an MPN-related thrombocytosis. Eligible subjects will include those who have not been previously treated for thrombocytosis or have not received platelet-lowering therapy for at least 2 weeks prior to study treatment. Each subject will receive anagrelide CR at a starting dose of 0.5 mg b.i.d. (1.0 mg/day), and is anticipated to continue study treatment for at least 24 weeks. Subjects who have achieved clinical benefit in the opinion of the Investigator and who are tolerating the study drug may continue study treatment until they develop unacceptable toxicity or other discontinuation criteria have been met.
The primary efficacy endpoint will be the proportion of subjects who achieve a platelet response (CR or PR). The safety and tolerability of study treatment will be assessed by the frequency and severity of adverse events as determined by NCI Common Terminology Criteria for Adverse Events (CTCAE) v 4.03. The PK profile of anagrelide CR will be assessed at the initial (0.5mg b.i.d.) and final titrated dose levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Anagrelide CR (GALE-401) | Experimental | Drug: Anagrelide CR |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Provide written informed consent prior to any study specific procedure
- Male or female patients aged ≥ 18 years
- Diagnosis of a myeloproliferative neoplasm (i.e., chronic myelogenous leukemia (CML), polycythemia vera (PV), primary myelofibrosis (PMF), essential thrombocythemia (ET)) as determined by the treating physician, such as based on the 2008 World Health Organization (WHO) classification of myeloid malignancies
- Baseline platelet count ≥600 x 10e9/L as determined on two occasions at least 14 days apart prior to the first dose of study drug
- Requirement for platelet reduction therapy as assessed by the Investigator
Key exclusion criteria (13 listed)
- Other MPN diagnoses not specifically included above: Chronic neutrophilic leukemia, chronic eosinophilic leukemia, mastocytosis, and unclassifiable MPNs
- Previously found to be refractory to anagrelide therapy (i.e., failure to achieve a platelet count <600 x 10e9/L for reasons other than anagrelide-related toxicity)
- History of coronary artery disease requiring a revascularization procedure within 3 months prior to screening
- Left bundle branch block or sustained ventricular tachycardia (>30 seconds) evident on 12-lead ECG at screening
- Tachycardia defined as resting heart rate >100 bpm at screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Anagrelide CRAlso: GALE-401Starting dose of 0.5 mg b.i.d. (1.0 mg/day) titrated at weekly intervals, on an individual basis, to achieve the lowest dose required to achieve and maintain a target platelet count of 150 - 400 x 10e9/L, tolerability permitting. The dose will be increased at weekly intervals in steps not exceeding 0.5 mg/day; the rate of dose titration may be reduced (i.e., up to once every 2 weeks) at the discretion of the Investigator. | Drug | Anagrelide CR (GALE-401) |
Primary outcome
Platelet Response
Time frame: Up to 24 weeks
The primary efficacy endpoint will be the proportion of subjects who achieve a platelet response (complete or partial response), with response defined according to the following criteria: * Complete response (CR): platelet count of ≤400 x 10e9/L maintained for at least 4 weeks * Partial response (PR): a platelet count of ≤600 x 10e9/L or a ≥50% reduction from baseline and maintenance of the reduction for at least 4 weeks * Nonresponse: failure to meet CR or PR criteria
2 secondary outcomes
Number of subjects with adverse events
Time frame: Throughout study treatment (expected average of 12 months)
Plasma concentrations of anagrelide
Time frame: Up to 13 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Anagrelide CR in people with Thrombocytosis and Myeloproliferative Neoplasms, run by Galena Biopharma, Inc. at 11 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Platelet Response (time frame: Up to 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2017; AzTrials last refreshed its copy on 6 Nov 2026.